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Drugs. 2021 Jun;81(8):953-956. doi: 10.1007/s40265-021-01520-2.
Fosdenopterin (Nulibry) is a synthetic cyclic pyranopterin monophosphate that is being developed by Origin Biosciences (a subsidiary of BridgeBio Pharma) for the treatment of molybdenum cofactor deficiency (MoCD) type A. Fosdenopterin was recently approved by the US FDA for use in reducing the risk of mortality in paediatric and adult patients with MoCD type A. This article summarizes the milestones in the development of fosdenopterin leading to this first approval.
福司地诺贝特（Nulibry）是一种合成的环状吡喃并喋呤单磷酸酯，由 Origin Biosciences（BridgeBio Pharma 的子公司）开发，用于治疗钼辅酶缺陷型 A（MoCD 型 A）。福司地诺贝特最近获得美国 FDA 批准，用于降低 MoCD 型 A 儿科和成年患者的死亡率。本文总结了福司地诺贝特开发过程中的重要里程碑，最终促成了该药的首次批准。
